It seems almost counterintuitive to assert that we have fundamental flaws in our cancer care delivery system at the same time that new drugs with improved outcomes seem to get FDA approval almost weekly. The major cancer conferences are filled with exciting presentations that change clinical practice overnight, with major advances in cancer immunotherapy, targeted therapy, and precision oncology happening with increasing frequency over the past 5-10 years.
Research institutes and smaller biotech firms, where much of the translational research on these breakthrough therapies occurs, have never seen such fertile ground. This is based on a combination of advances in genomics, immune-oncology, targeted therapies, various medical technologies, and a new, more progressively led FDA.
Yet the U.S.'s current care delivery structure, institutions, and, most of all, reimbursement system have not kept pace with the scientific advances. As clinicians, we have learned a new term, one without precedent in our training or prior professional experience: financial toxicity.
Health care reform and ongoing market forces have led to "perverse" incentives that can prevent a patient, even when insured, from receiving potentially lifesaving treatment for the simple reason that they can't afford their personal financial obligation for the drug, device, hospitalization, or all of the above. Detailing the roots, extent or possible solutions to this problem are well beyond this article, but nobody currently involved in the health care system will dispute its existence and its ongoing effect on cancer care.
The Breakthrough Oncology Accelerator was formed in October 2018. This is a unique, industry-wide partnership that leverages the collective strengths and wisdom from among the translational research, clinical, and financial innovators in these newer, breakthrough treatments. Starting with the Translational Genomics Research Institute (TGen), Origin Commercial Ventures, and Atlantic Health System, the Accelerator is designed to improve patient access to lifesaving therapies through more rapid deployment of new research trials and novel payment mechanisms post-approval.
With Atlantic Health as the first clinical site, the Accelerator is expanding, providing access to those health systems whose desire is to ensure patient access to CAR-T and the most promising cancer care treatments during clinical/translational investigation and post-agency approval.
How is the breakthrough oncology accelerator currently helping patients?
The Accelerator has opened multiple early and late-phase clinical trials at Atlantic Health, currently available at only a few centers around the world. In February 2019, it will also be opening a first of its kind, dedicated high-promise therapeutics center at Morristown Medical Center.
In partnership with several research and industry organizations, the purpose is to further extend the breadth of the best and brightest ideas in translational medicine, cancer care, and finance-and ultimately ensure that these ideas become reality, reaching patients throughout the world.
How will the breakthrough oncology accelerator change cancer care?
The next 25 years represent the dawn of high promise therapeutics, or those breakthrough therapies that have the opportunity to cure complex cancers with only one or a limited number of treatments.
This era of high promise will also be accompanied by high complexity and high cost, both in research and development and then also post-approval. We have seen the start of this over the previous 2 decades as more precise genomic characterization of cancer has enabled more customization of cancer research strategies.
The economic risks and amount of capital required by our new modes of translational research have become too great to manage under a typical investor model. This will require more substantial and direct funding for commercialization programs at research institutes, treating the institutes and their scientific leadership more as co-investors, with sufficient autonomy and control to more independently guide priorities, streamline development, and de-risk therapies for the end-investors: pharma and more traditional investors.
Although we see this as similar to the adaptive financing techniques practiced by venture capitalists, we believe the institutes and their start-ups should control much more of the funding and decision making over a longer period, based on their intimate understanding of the science, know-how, and protocols required to make breakthroughs a reality.
Once a drug or device is FDA-approved, a new set of problems arise related to safe administration of complicated therapies and, as mentioned, potential financial toxicity related to the high cost of these treatments and the failure of the current reimbursement system to adequately anticipate these high-cost, high-reward interventions in a manner that ensures patient access without "breaking the bank."
For example, the global cancer immunotherapy market is expected to reach $120 billion by 2021, yet much of the provider community lacks the know-how, infrastructure, and resources to deploy such therapies. For many reasons, the costs of these therapies have ballooned to more than 900 percent of traditional standards of care. With additional FDA approvals, more patients become eligible for these expensive yet effective treatments. Can our current system absorb the cost?
Yet the best example in today's cancer therapy landscape is CAR-T or chimeric antigen receptor T-cell therapy, just approved for two malignant hematologic indications in 2018. With hundreds of clinical trials currently open exploring a wide variety of indications for variations on CAR-T, it is likely that there will soon be additional approvals, and the market is expected to grow by 32.5 percent over the next several years (see Figures 1 and 2).
This growth is not without issues: the high cost and complexity of CAR-T therapy has greatly limited its adoption and availability thus far. A single patient's bill is reported to be several hundred thousand dollars, if not more. All components of the health care ecosystem-patients, physicians, hospitals, payers, government, and pharma-are rightfully concerned about cost versus outcome. How do we balance the lifesaving potential of a single treatment against the life-altering cost of the therapy?
For this expected growth rate in CAR-T to materialize, a truly multifaceted solution has to prevail-one that takes into account the multiple barriers in place and can manage them in a nimble, dynamic way including development, access, cost, and consistency (Figure 3).
Multiple solutions have been proposed, mostly focused on cost of care. However, this is unlikely to be a long-term solution, as future CAR-T-based therapies are likely to be even more expensive to pre-clinically develop and produce post-approval in a commercial setting. Figure 4 outlines some of the proposed models.
The Breakthrough Oncology Accelerator solution to these issues can best be described as an expanding network of clinical partners and finance vehicles, focused on accelerating the best and brightest ideas for breakthrough cancer therapies and providing patients with faster access.
Figure 5 outlines the features of the Accelerator, listing the needs of the relevant participants on the left, connecting them by the "transaction" (care provided), and updating how their needs are addressed in the right-hand column.
The Accelerator serves as intermediary between the health care components (patient, physician, hospital, and pharma) and the financial components (private, government payer), assuring all entities maintain clinical and operational excellence while minimizing financial risk on either end. Thus, by providing ready-made access to funding sources optimized for large-scale cancer care transactions, the Accelerator ensures ongoing access to CAR-T therapy for patients in need (See Figure 5).
As shown in Figure 6, the Accelerator is selecting experienced regional CAR-T centers of excellence, which will be accepted by the financing arm as the premier places to be treated with CAR-T therapy, facilitating acceptable payments throughout the health care ecosystem and ensuring adequate patient access to care. Going forward, this same model can be sequentially applied to future novel therapies, as developed and approved.
Looking Forward
Atlantic Health System and its partners, TGen and OriginCV, are excited to have created the Breakthrough Oncology Accelerator and to make it operational at AHS facilities in 2018.
We continue to evaluate clinical trial opportunities and open trials that make sense for our clinical expertise and patient populations, improving patient access to novel therapies.
Ongoing financial structuring and logistical planning will enable the Accelerator to soon implement advanced payment and reimbursement structures, ensuring our patients retain access to CAR-T therapy and other, to be developed, new treatments post-approval.
Eric D. Whitman, MD, FACS, is Medical Director of Atlantic Health System Cancer Care, and Founding Member of the Breakthrough Oncology Accelerator.